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PRX-102 /kg every 2 weeks

Phase 2

Fabry Disease | Small molecule | Metabolic |Icon Plc|Last Updated: Mar 19, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDDMCBiomarker
Total Trials2
Total Enrollment38
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06328608A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry DiseasePHASE2 RECRUITING 22 — — Jul 29, 2025Apr 1, 2031Mar 19, 202612 United States, Austria +4
NCT05710692Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry DiseasePHASE2 RECRUITING 16 — — Aug 1, 2023Aug 1, 2029Mar 18, 202610 Japan
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Study Endpoints
Primary Endpoints
Incidence of Treatment Emergent Adverse Events (TEAEs)
12 Months
Incidence of Infusion Related Reactions (IRRs)
12 Months
Incidence of Injection site reactions (ISRs)
12 Months
Change in Tanner stage
Baseline and 12 Months

Tanner Staging of Sexual Development will be used to assess sexual development (i.e. breast development (B1 to B5) and pubic hair development (Ph-1 to Ph-5) in females and pubic hair and genetical development (G1-G5) in males.

Change from baseline of 12-lead ECG quantitative parameters: Mean Heart Rate
Baseline and 12 Months
Change from baseline of 12-lead ECG quantitative parameters: PR Interval
Baseline and 12 Months
Change from baseline of 12-lead ECG quantitative parameters: QRS Duration
Baseline and 12 Months
Change from baseline of 12-lead ECG quantitative parameters: QT Interval
Baseline and 12 Months
Change from baseline of 12-lead ECG quantitative parameters: QTc Interval
Baseline and 12 Months
Change from baseline of 12-lead ECG quantitative parameters: ST Segment
Baseline and 12 Months
Incidence of treatment-emergent Anti-Drug Antibodies (ADAs)
Baseline and 12 Months
Incidence of premedication use at each visit and change of infusion premedications from baseline
Baseline and 12 Months
Pharmacokinetics: Time to maximum plasma concentration (tmax)
Stage I - Baseline, week 2, week 4, week 12, week 26 and week 52; Stage II -Baseline, week 12, week 26 and week 52]
Pharmacokinetic : Area under the plasma concentration-time curve from time 0 to time t (AUC0 t)
Stage I - Baseline, week 2, week 4, week 12, week 26 and week 52; Stage II -Baseline, week 12, week 26 and week 52]
Pharmacokinetics: Area under the curve from time 0 to 2 weeks (AUC0-2wk)
Stage I - Baseline, week 2, week 4, week 12, week 26 and week 52; Stage II -Baseline, week 12, week 26 and week 52]
Pharmacokinetics: Area under the curve from time 0 to infinity (AUC0-∞)
Stage I - Baseline, week 2, week 4, week 12, week 26 and week 52; Stage II -Baseline, week 12, week 26 and week 52]
Pharmacokinetics: Terminal half-life (t1/2)
Baseline, week 2, week 4, week 12, week 26 and week 52]
Pharmacokinetics: Area under the curve over a dosing interval (AUCτ)
Baseline, week 2, week 4, week 12, week 26 and week 52; Stage II -Baseline, week 12, week 26 and week 52]
Pharmacokinetics: Observed drug concentration at the end of the dosing interval (Cτ)
Stage I - Baseline, week 2, week 4, week 12, week 26 and week 52; Stage II -Baseline, week 12, week 26 and week 52]
Pharmacokinetics: Clearance (Cl)
Stage I - Baseline, week 2, week 4, week 12, week 26 and week 52; Stage II -Baseline, week 12, week 26 and week 52]
Pharmacokinetics: Volume of distribution (Vz)
Stage I - Baseline, week 2, week 4, week 12, week 26 and week 52; Stage II -Baseline, week 12, week 26 and week 52]
Change in eGFR
Baseline and 12 Months
Change in annualized eGFR slope
Baseline and 12 Months
Change in urine albumin levels
Baseline and 12 Months
Change in urine protein levels
Baseline and 12 Months
Change from baseline in LVMi as assessed by echocardiogram
Baseline and 12 Months

Echocardiogram parameters include left ventricular mass index (LVMi)

Incidence of any cardiac arrythmias as assessed by Holter ECG
Baseline and 12 Months
Change in plasma levels of cardiac biomarkers
Baseline and 12 Months

High-sensitivity cardiac troponin T (hs-cTnT) and N- terminal pro brain natriuretic peptide (NT-Pro BNP) will be assessed.

Change in plasma level of Gb3 concentration (nM)
Baseline and 12 Months
Change in plasma level of lyso-Gb3 (nM)
Baseline and 12 Months
Change in urine level of lyso-Gb3 (nM)
Baseline and 12 Months
Incidence of change from baseline in the number of different pain medications
Baseline and 12 Months
Incidence of Fabry Clinical Events
12 Months

FCEs are classified into four categories: renal, cardiac, cerebrovascular and death due to non-cardiac reasons

Change from baseline of Mainz Severity Score Index (MSSI) scores
Baseline and 12 Months

Domains (general, neurological, cardiovascular, renal dysfunction)

Change from baseline of PedsQL-GI (or GSRS for subjects who reaches 18 yrs of age) scores
Baseline and 12 Months
Change from baseline of FPHPQ scores
Baseline and 12 Months
Change from baseline of PedsQL-PPQ (or BPI-SF for subjects who reaches 18 yrs of age) scores
Baseline and 12 Months
Change from baseline of EQ-5D-Y (or EQ-5D-5L for subjects who reaches 18 yrs of age) scores
Baseline and 12 Months
Change of laboratory tests' results
12 Months, 24 Months and through study completion (an average of 4.5 years)
Change in in body weight in kilograms
12 Months, 24 Months and through study completion (an average of 4.5 years)
Change in height in centimeters
12 Months, 24 Months and through study completion (an average of 4.5 years)
Change from baseline of 12-lead ECG quantitative parameters: Mean Heart Rate, PR Interval, QRS Duration, QT Interval, QTc Interval, and ST Segment
12 Months, 24 Months and through study completion (an average of 4.5 years)

Quantitative ECG parameters will be summarized by cohort and overall

ADA status change from baseline
12 Months, 24 Months and through study completion (an average of 4.5 years)
Change from baseline of Maximum plasma concentration (Cmax), pharmacokinetic parameter
Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)
Change from baseline of Time to maximum plasma concentration (tmax), pharmacokinetic parameter
Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)
Change from baseline of Area under the plasma concentration-time curve from time 0 to time t (AUC0 t), pharmacokinetic parameter
Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)
Change from baseline of Area under the curve from time 0 to 2 weeks (AUC0-2wk), pharmacokinetic parameter
Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)
Change from baseline of Area under the curve from time 0 to infinity (AUC0-∞), pharmacokinetic parameter
Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)
Change from baseline of Terminal half-life (t1/2), pharmacokinetic parameter
Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)
Change from baseline of Area under the curve over a dosing interval (AUCτ), pharmacokinetic parameter
Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)
Change from baseline of Observed drug concentration at the end of the dosing interval (Cτ), pharmacokinetic parameter
Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)
Change from baseline of Clearance (Cl), pharmacokinetic parameter
Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)
Change from baseline of Volume of distribution (Vz), pharmacokinetic parameters
Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Single Arm - Pegunigalsidase alfa (PRX-102)EXPERIMENTALFor Cohort C, PXR-102 administered every two weeks at 1.0 mg/kg is believed to be the minimum effective dose. For Cohorts A and B, the starting dose will be 1.0 mg/kg every two weeks but it may be adjusted on the outcomes of Stage I, with the support of the Data Safety Monitoring Board.
PRX-102 1 mg/kg every 2 weeks or PRX-102 2 mg/kg every 4 weeksEXPERIMENTALPRX-102 1 mg/kg every 2 weeks or PRX-102 2 mg/kg every 4 weeks (available only in the optional extension part)
Interventions
NameTypeDescription
PRX-102 1 mg/kg every two weeksDRUGDrug: PRX-102 1 mg/kg every two weeks
PRX-102 1 mg/kg every 2 weeksDRUGPRX-102 1 mg/kg every 2 weeks
PRX-102 2 mg/kg every 4 weeksDRUGPRX-102 2 mg/kg every 4 weeks
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Eligibility Criteria
Age Range2 Years — 17 Years
SexALL
Healthy VolunteersNo
Study Sites12

Inclusion Criteria: * Participants with the provision of informed consent from their legal guardians * Boys and girls aged 2 to 7 years (Cohort A), 8 to 12 years (Cohort B), or 13 to \<18 years (Cohort C). * Confirmed diagnosis of Fabry disease * Presence of at least one of the following characteri...

Countries:United StatesAustriaFranceNorwaySpainUnited KingdomJapan
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05710692primaryCompletionDate: changed
LOWMay 26, 2026NCT06328608primaryCompletionDate: changed
LOWMay 24, 2026NCT05710692studyFirstPostDate: changed
LOWMay 24, 2026NCT06328608studyFirstPostDate: changed